Applying Evidence Standards to Guide Gene Therapy Use in BCG-Unresponsive NMIBC

EP: 1.Evolving Guidelines: Impact of Gene Therapy and BCG Failure Redefinitions on High-Risk NMIBC Patient Management

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EP: 2.Applying Evidence Standards to Guide Gene Therapy Use in BCG-Unresponsive NMIBC

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High costs of gene therapies necessitate robust evidence standards to guide clinical use and coverage determinations for patients with BCG-unresponsive non-muscle invasive bladder cancer (NMIBC). The primary evidence foundation relies on Phase 3 trial data demonstrating complete response rates at three months and durability of response over time. Intention-to-treat analysis provides the most meaningful efficacy data, showing disease-free survival rates at one and two years, which are crucial metrics for both clinical decision-making and payer coverage decisions.

Comparative effectiveness research remains challenging due to the absence of head-to-head randomized trials between different gene therapies. This limitation makes it difficult to determine which agents offer superior efficacy or tolerability profiles. Real-world evidence studies help bridge this gap by validating clinical trial results in broader patient populations, providing reassurance that trial outcomes translate to routine clinical practice. These studies are essential for building confidence in treatment selection and sequencing strategies.

Health economic analyses will increasingly influence coverage decisions and treatment pathways. Understanding which therapies can reduce healthcare system costs through fewer hospitalizations, avoided cystectomies and reduced treatment intensity will be crucial for long-term sustainability. The integration of efficacy data, real-world evidence and economic outcomes will ultimately shape standardized treatment protocols and reduce unwarranted variation in clinical practice across different healthcare systems.